Strongly


An expression of the live unlimited spirit, stories and voices of the MDA community

Five Questions with ALS Researcher Rita Sattler

Rita Sattler, associate professor of neurobiology at Barrow Neurological Institute, Dignity Health – St. Joseph’s Hospital in Phoenix, was awarded an MDA research grant totaling $300,000 over three years to elucidate the mechanisms underlying ALS (amyotrophic lateral sclerosis) caused by expansions in the C9ORF72 gene. Please describe your current research. The goal is to study . . .

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Wyoming State Goodwill Ambassador – Terra McCumber

Terra McCumber, Age 9 Bar Nunn, WY Home and Family Terra lives with her mom and stepdad, Darshee and Derek Buist, her brother Anthony, and stepbrothers Xavier and Zayden. Diagnosis Terra is diagnosed with Limb-Girdle Muscular Dystrophy. Limb-Girdle muscular dystrophy isn’t one disease. It’s a group of disorders affecting voluntary muscles, mainly those around the . . .

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Five Questions with SBMA Researcher Diane Merry

Diane Merry, associate professor at Thomas Jefferson University in Philadelphia, is working to identify therapeutic opportunities to promote normal androgen receptor function while preventing the toxic effects of polyglutamine expansion in spinal-bulbar muscular atrophy (SBMA). With GlaxoSmithKline, Merry will test small molecule compounds that activate an enzyme called SIRT1 in cell and mouse models of . . .

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PTC Announces Phase 3 Trial Results for Ataluren in DMD

Background: PTC Therapeutics yesterday announced results from the phase 3 “ACT DMD” trial of ataluren, an oral, first-in-class, protein restoration therapy for the treatment of nonsense mutation Duchenne muscular dystrophy (DMD). The trial results showed clinically meaningful benefits for ataluren-treated patients, indicating the drug may slow functional decline in DMD patients, with some patients apparently . . .

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Five Questions with FSHD Researcher Antoine de Morrée

Antoine de Morrée, a postdoctoral scholar at Palo Alto Veterans Institute for Research and Stanford University in Palo Alto, Calif., is testing a way to stop muscles from making toxic DUX4 protein as a possible treatment forfacioscapulohumeral muscular dystrophy (FSHD). The goals of de Morrée’s work are to understand how cells make stabile RNA messages, . . .

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Sarepta Reports Data from Phase 2 Trial of Eteplirsen for Treatment of DMD

Background: Sarepta Therapeutics reported today additional long-term clinical efficacy and safety data from its pivotal phase 2b program of eteplirsen to treat Duchenne muscular dystrophy (DMD). The data demonstrated that over a three-year period of time, trial participants who were treated with eteplirsen experienced a slower rate of decline and were, on average, able to . . .

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Enrollment in Diaphragm Pacing Systems for People with ALS Halted

In September, the Data Safety and Monitoring Board (DSMB), constituted to oversee the Diaphragm Pacing System in Participants with Amyotrophic Lateral Sclerosis (DPS in ALS) study, being conducted in the United States, issued a recommendation that new enrollment be halted. At the same time, the DSMB recommended that patients already randomized to the diaphragm pacing . . .

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Sarepta Therapeutics Announces USPTO Decision in Patent Interference Case with BioMarin Pharmaceutical

– Importance of USPTO decision not ascertainable until determinations are rendered in two remaining composition interferences and planned appeal – CAMBRIDGE, Mass.–(BUSINESS WIRE)–Sep. 30, 2015– Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced that the Patent Trial and Appeal Board (PTAB) of the United States Patent and Trademark Office (USPTO) has . . .

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Five Questions with Researcher Criss Hartzell

Criss Hartzell, professor of cell biology at Emory University School of Medicine in Atlanta, Ga., recently was awarded an MDA research grant to study the ANO5 gene. Mutations in the gene have been implicated in both type 2L limb-girdle muscular dystrophy (LGMD) and a subtype of Miyoshi distal muscular dystrophy (a type of distal muscular dystrophy, . . .

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